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Nesolicaftor

Nesolicaftor, also known as PTI-428, is a promising drug being studied in clinical trials for the treatment of cystic fibrosis (CF). These trials aim to evaluate the safety and effectiveness of Nesolicaftor when used in combination with other drugs (Dirocaftor and Posenacaftor) in adults with CF who have rare genetic mutations. The studies are designed to help researchers understand how well this drug combination works in improving lung function and other important aspects of CF management.

Table of Contents

What is NESOLICAFTOR?

NESOLICAFTOR, also known as PTI-428, is a promising new medication being studied for the treatment of cystic fibrosis (CF). Cystic fibrosis is a genetic disorder that affects the lungs, pancreas, and other organs, causing thick, sticky mucus to build up and leading to various health problems.[1] [2]

NESOLICAFTOR is part of a class of drugs called CFTR modulators. These medications work by targeting the underlying cause of cystic fibrosis at the cellular level. NESOLICAFTOR is being studied in combination with two other drugs, dirocaftor and posenacaftor, as a potential treatment for CF patients with rare genetic mutations.

How Does NESOLICAFTOR Work?

NESOLICAFTOR is designed to help improve the function of the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) protein. In people with CF, this protein is either missing or doesn’t work properly, leading to the buildup of thick mucus in various organs. By enhancing CFTR protein function, NESOLICAFTOR aims to reduce mucus buildup and improve symptoms in CF patients.[1] [2]

Clinical Trials

NESOLICAFTOR is currently being studied in clinical trials to evaluate its safety and effectiveness. Two important studies are underway:

  1. A Phase III trial called “CHOICES” is evaluating the combination of dirocaftor, posenacaftor, and NESOLICAFTOR in adults with CF aged 18 years or older. This study aims to assess the efficacy and safety of the drug combination after 8 weeks of treatment.[1]

  2. A Phase IIb trial, also called “CHOICES,” is studying the same drug combination in adults with CF aged 18 years or older. This trial is specifically looking at patients with rare CFTR mutations.[2]

Both trials are randomized, double-blind, and placebo-controlled, which means that participants are randomly assigned to receive either the study medication or a placebo, and neither the participants nor the researchers know who is receiving which treatment. This design helps ensure the most accurate and unbiased results.

Eligibility Criteria

To participate in these clinical trials, patients must meet certain criteria. Some key eligibility requirements include:

  • Age 18 years or older
  • Confirmed diagnosis of cystic fibrosis
  • Clinically stable CF disease
  • Forced expiratory volume in one second (FEV1) between 40% and 90% of predicted value (FEV1 is a measure of lung function)
  • Body mass index (BMI) between 16 kg/m² and 30 kg/m²
  • Non-smoker and non-tobacco user

It’s important to note that patients with certain common CF mutations or those currently taking other CFTR modulators may not be eligible for these specific trials.[1] [2]

Potential Benefits

The clinical trials are evaluating several potential benefits of NESOLICAFTOR in combination with dirocaftor and posenacaftor. These include:

  • Improved lung function, measured by percent predicted forced expiratory volume in 1 second (ppFEV1)
  • Reduced sweat chloride levels (a diagnostic marker for CF)
  • Increased body weight
  • Improved quality of life, as measured by the Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain

These outcomes are being assessed after 4, 6, and 8 weeks of treatment to determine the effectiveness of the medication.[1] [2]

Safety and Side Effects

As with any new medication, safety is a crucial aspect of the clinical trials for NESOLICAFTOR. The studies are closely monitoring for any potential side effects or adverse events. Safety assessments include:

  • Monitoring of treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
  • Regular clinical laboratory tests (blood tests, urine tests)
  • Physical examinations
  • Electrocardiography (ECG) to monitor heart function
  • Vital signs measurements

Additionally, the trials are studying how the body processes NESOLICAFTOR by measuring drug levels in the blood (pharmacokinetics).[2]

Future Prospects

NESOLICAFTOR, in combination with dirocaftor and posenacaftor, represents a potential breakthrough in the treatment of cystic fibrosis, especially for patients with rare genetic mutations. If the clinical trials show positive results, this medication could offer new hope for CF patients who have limited treatment options.

It’s important to remember that NESOLICAFTOR is still in the research phase, and more studies may be needed before it becomes available as a approved treatment. Patients with CF should continue to work closely with their healthcare providers to manage their condition and stay informed about new treatment options as they develop.[1] [2]

Aspect Details
Drug Name Nesolicaftor (PTI-428)
Study Type Phase IIb and Phase III clinical trials
Study Design Randomized, double-blind, placebo-controlled, crossover study
Participant Age 18 years or older
Primary Endpoint Change in percent predicted FEV1 after 8 weeks of treatment
Secondary Endpoints Sweat chloride levels, body weight, quality of life (CFQ-R), safety and tolerability
Dosage Maximum daily dose of 10 mg
Administration Route Oral (capsule)
Treatment Duration Up to 16 weeks
Key Inclusion Criteria Confirmed CF diagnosis, FEV1 40-90% predicted, BMI 16-30 kg/m²
Key Exclusion Criteria Common CFTR mutations, current use of CFTR modulators

FAQ

  • What is Nesolicaftor and how does it work? Nesolicaftor (PTI-428) is a drug being studied for the treatment of cystic fibrosis. It works by targeting the CFTR protein, which is faulty in people with CF. The goal is to improve the function of this protein and help manage CF symptoms.
  • Who can participate in these clinical trials? The trials are open to adults (18 years or older) with cystic fibrosis who have rare CFTR mutations. Participants must meet specific criteria, including having stable CF disease and a certain range of lung function (FEV1 between 40% and 90% of predicted).
  • What is the main goal of these clinical trials? The main goal is to evaluate how well Nesolicaftor, when combined with Dirocaftor and Posenacaftor, improves lung function in CF patients with rare mutations. This is measured by changes in forced expiratory volume in one second (FEV1) after 8 weeks of treatment.
  • What other effects of Nesolicaftor are being studied? Besides lung function, the trials are looking at other important factors such as changes in sweat chloride levels, body weight, quality of life (using a questionnaire), and overall safety and tolerability of the drug combination.
  • How long do these clinical trials last? The trials have different phases, but the primary evaluation period is 8 weeks. Some parts of the study may extend up to 24 weeks to assess the long-term effects and safety of the treatment.

Ongoing Clinical Trials on Nesolicaftor

  • Study on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Denmark Germany Italy The Netherlands +3

  • Study on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis

    Not recruiting

    1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia France Germany Italy The Netherlands +3

Glossary

  • Cystic Fibrosis (CF): A genetic disorder that affects the lungs, pancreas, and other organs, causing thick, sticky mucus to build up and leading to various health problems.
  • CFTR: Cystic Fibrosis Transmembrane Conductance Regulator, the protein that is faulty in people with CF and is targeted by drugs like Nesolicaftor.
  • FEV1: Forced Expiratory Volume in 1 second, a measure of lung function that shows how much air a person can forcefully exhale in one second.
  • Sweat chloride test: A diagnostic test for CF that measures the amount of salt (chloride) in a person's sweat. High levels can indicate CF.
  • Randomized, Double-Blind, Placebo-Controlled: A study design where participants are randomly assigned to receive either the study drug or a placebo, and neither the participants nor the researchers know who is receiving which treatment.
  • Crossover study: A type of clinical trial where participants receive different treatments in a specific order, allowing each person to serve as their own control.
  • Rare CFTR mutations: Uncommon genetic changes in the CFTR gene that cause cystic fibrosis but are less frequent than more common mutations like F508del.
  • Organoid response: A method of testing how well CF drugs might work for an individual by using tiny, lab-grown versions of their organs (organoids).
  • CFQ-R: Cystic Fibrosis Questionnaire-Revised, a tool used to measure quality of life in people with CF.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including how it's absorbed, distributed, metabolized, and eliminated.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effects-of-dirocaftor-posenacaftor-and-nesolicaftor-for-adults-with-cystic-fibrosis/
  2. http://clinicaltrials.eu/trial/study-on-the-effects-of-dirocaftor-posenacaftor-and-nesolicaftor-for-adults-with-cystic-fibrosis-2/